Articles tagged with Cystic Fibrosis
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Scientists successfully created airway basal stem cells in vitro from induced pluripotent stem cells, which may lead to new treatments for airways and lungs damaged by various diseases. The study's findings could also enable the development of disease models and targeted drug approaches.
A genotype-agnostic gene therapy for cystic fibrosis has shown promise in clinical trials, potentially treating the disease in any patient, independent of their underlying mutation. Challenges remain to be overcome, including developing effective drug delivery systems that can reach pulmonary epithelial cells at low doses.
A major national study found that oral antibiotics are as effective as intravenous antibiotics in killing Pseudomonas aeruginosa, a common germ causing chronic lung infection in CF patients. This means CF patients with a new infection can be treated at home and saved from hospital admissions.
Researchers discovered that a urine test of bicarbonate can measure CFTR function in patients with cystic fibrosis. This test may predict the effectiveness of new treatments for the disease.
The Vav3 protein creates bacterial docking stations on airways' surface, facilitating recurrent infections in cystic fibrosis. Inhibiting this protein may prevent respiratory complications by limiting bacterial adhesion.
Researchers have developed a radical non-invasive X-ray technology that can pinpoint localised areas of deficiency in lungs, improving diagnoses and treatments for cystic fibrosis. The technology, called X-ray velocimetry, measures airflow through the lungs during breathing cycles, enabling better assessment of lung function.
Researchers have discovered a new disease that affects the same chloride channel as cystic fibrosis, potentially leading to improved treatment options. The study found that patients with this disease do not develop respiratory symptoms, suggesting a possible treatment target for cystic fibrosis.
Scientists have revealed the first known atomic structure of a molecular machine responsible for installing critical signaling proteins into cellular membranes. The study provides new insights into how membrane proteins become established in membranes and how they change from their unstructured form to their functional form.
A randomized trial found that extended rituximab infusions were associated with lower relapse rates in patients with antineutrophil cytoplasmic antibody-associated vasculitis. Long-term rituximab use is considered an effective and probably safe treatment option for patients with AAV, according to the study's findings.
Researchers compared federal and foundation funding for sickle cell disease and cystic fibrosis, finding that higher funding was associated with increased research productivity and faster drug development. The study suggests that increasing funding could improve treatment options for these two diseases.
Researchers found that using plant-based analogs of cholesterol improves the shape and structure of nanoparticles, allowing them to deliver genes more effectively. This boosts delivery by up to 10-fold and enables patients to be treated with higher efficacy.
A multi-center study has identified a research-based ultrasound screening method that can predict which children with cystic fibrosis are at higher risk for advanced liver disease. The test shows promise in identifying patients who could benefit from targeted therapies to prevent the development of this life-threatening condition.
Researchers at Monash University have developed a new technology to visualize airflow in the lungs, enabling faster and more accurate diagnoses. The technology can pinpoint localized areas of deficiency and has potential for treating respiratory illnesses earlier.
Scientists aim to eliminate persister cells, causing chronic health issues like airway infections and tuberculosis, by targeting their formation mechanisms through self-digestion. By mapping the self-digestion-related mechanisms in E. coli, researchers hope to develop effective anti-persister therapeutics.
Researchers have developed a new treatment that completely kills the bacterial infection causing severe decline in lung function and death in cystic fibrosis patients. The treatment combines three antibiotics with no side effects, improving quality of life and survival chances for infected CF patients.
Researchers found that people with one mutated copy of the cystic fibrosis gene, known as CF carriers, are at higher risk for various CF-related conditions, including pancreatitis and type 1 diabetes. The study's results suggest a substantial burden of illness from these conditions, impacting over 10 million Americans.
Researchers discovered genetic variations in RNF5 dampen CFTR protein expression, potentially reducing lung infections. This study offers a new approach to understanding cystic fibrosis and developing targeted treatments.
A new treatment approach for cystic fibrosis may reduce the need for lung transplants and lower mortality rates. Researchers found that a small molecule reduced inflammation and cleared lungs of bacteria in a laboratory model.
A rapid test for cystic fibrosis has been developed using a 2-minute swiping process that analyzes compounds in perspiration samples, with 98% specificity and 96% sensitivity. The test uses desorption electrospray ionization mass spectrometry and machine learning to identify cases.
A study in eLife reveals how respiratory bugs in cystic fibrosis patients interact, influencing disease progression and survival. The research found that one bacterium enhances the movement of another, while others significantly increase its mobility.
A three-drug combination improved lung function and reduced symptoms in patients with the most common genetic mutation for cystic fibrosis. The therapy, approved by the FDA, targets the underlying cause of the disease and demonstrated significant benefits in lung function and quality of life.
Researchers have identified the oldest known ortholog of the ion channel defective in cystic fibrosis, found in ancient sea lampreys approximately 450 million years ago. The protein diverges significantly from its human counterpart and has unique functional properties, suggesting a distinct evolutionary history.
Researchers at the University of Iowa have made significant breakthroughs in delivering gene-editing proteins to airway cells without causing harm. The new peptide-based platform shows promise in treating diseases like cystic fibrosis, COPD, and asthma by repairing or modifying disease-causing mutations.
Researchers found that cystic fibrosis carriers have a higher likelihood of experiencing bile duct obstruction and other digestive system phenotypes. The study used large-scale genomic data from the UK Biobank to identify these associations, which may provide answers for carriers who were previously left without information.
A study of 845 CF patients found that lumacaftor-ivacaftor improves lung function, body weight, and reduces intravenous antibiotic treatment needs. However, nearly one in five patients discontinued treatment due to adverse events, highlighting the need for cautious interpretation.
The Lancet Respiratory Medicine published a series of papers on cystic fibrosis treatment, including new CFTR modulator drugs showing promise and improving patient outcomes. Additionally, a three-in-one inhaler therapy was found to improve lung function and reduce asthma attacks in patients with severe asthma.
A nationwide trial led by Drexel University is investigating the effectiveness of Acceptance and Commitment Therapy (ACT) delivered via telemedicine to reduce anxiety and depression among cystic fibrosis patients. The study aims to improve lung function and optimize mental and physical health outcomes.
A new study suggests that Pseudomonas aeruginosa thrives in cystic fibrosis lungs due to an abundance of succinate, a byproduct of cellular metabolism. This excess succinate fuels the growth of the bacteria and promotes chronic infections.
A new imaging tool provides detailed images of the nasal airways in cystic fibrosis patients, showing impaired mucus clearance and cilia dysfunction. The technique enables clinicians to diagnose diseases earlier and optimize treatment for individual patients.
Researchers at the University of Trento have developed a genome editing strategy to permanently correct two types of mutations that cause cystic fibrosis. The 'SpliceFix' technique uses Crispr-Cas to edit patient-derived organoids, showing high precision and efficacy.
Researchers found ivacaftor significantly reduces P. aeruginosa, Staphylococcus aureus and Aspergillus spp. infections in CF patients, with a 32% reduction in P. aeruginosa and 15% reduction in Staphylococcus aureus.
Researchers created a human pancreas on a chip that can mimic the human pancreas and potentially help find therapeutic measures to manage glucose imbalance in people with cystic fibrosis. The device was used to study CF-related diabetes and type 1/2 diabetes, opening new possibilities for disease research.
A recent study found that including new clinical variables in the lung allocation score improved its accuracy for identifying the sickest cystic fibrosis patients. The updated scoring system also helped patients with chronic obstructive pulmonary disease (COPD) by re-evaluating their pulmonary function testing.
A study published in the American Journal of Respiratory and Critical Care Medicine found that e-cigarette vaping with nicotine impairs ciliary beat frequency, dehydrates airway fluid, and makes mucus more viscous. This can lead to increased risk of chronic bronchitis and other lung diseases.
A researcher at Oregon State University has received a five-year, $3.3 million grant to develop a way for cystic fibrosis patients to get molecular treatment via an inhaler. The treatment would work on every patient, regardless of their genetic mutation, and could vastly improve existing therapeutic methods.
Certain bacteriophages exacerbate bacterial infections in cystic fibrosis patients, particularly with Pseudomonas aeruginosa. These phage-carrying bacteria are more resistant to standard antibiotics, highlighting potential therapeutic targets.
A new study has found that cystic fibrosis patients who have a common virus may experience faster disease progression. Cytomegalovirus infection was the most important factor linked to disease progression, with patients having the virus referred for lung transplants at a much younger age and dying ten years earlier on average.
Researchers found that the medication amphotericin helps lung cells function, making it easier for patients to fight chronic bacterial infections. The finding holds special promise for a subset of patients who do not respond to current treatments.
Researchers have found that an approved drug can function as a molecular prosthetic, restoring infection-fighting properties in lung tissue and treating cystic fibrosis. The approach bypasses defective proteins and could benefit all types of CFTR mutations.
Researchers developed a novel gene therapy approach that utilizes engineered transfer RNA (tRNA) molecules to suppress nonsense mutations. This technology has the potential to correct 10-15% of inherited genetic diseases, including Duchenne muscular dystrophy and cystic fibrosis.
A Phase 3 study found that lumacaftor/ivacaftor is safe and effective in treating cystic fibrosis in children aged 2-5 years. The treatment combination shows promise in arresting disease progression and improving long-term outcomes, including improved pancreatic function.
Chemists at McMaster University have identified new chemical signatures in a small amount of blood that could detect cystic fibrosis in infants earlier and more accurately. These biomarkers can be analyzed using mass spectrometry, reducing costs and anxiety for families.
Researchers identify strong genetic risk factor for IPF, linking mucociliary dysfunction to lung scarring. The study provides a breakthrough in understanding the cause of IPF and potentially its treatment.
Researchers identified distinct molecular signatures in children with cystic fibrosis, which may help predict disease severity and treatment response. The findings pave the way for precision medicine, allowing for therapies tailored to each patient's unique biology.
A study published in Nature Communications identifies extracellular DNA degradation as a key mechanism of lung inflammation induced by silica exposure. Treatment with DNase I may prevent silica-induced lung inflammation, suggesting a new therapeutic target for silicosis.
A new study has identified a specific peptide that directly binds to the ion-channel CFTR, impairing its function and triggering cellular stress and inflammation. This interaction can be inhibited by a potentiator of CFTR, which may offer protection against gluten-induced intestinal symptoms.
Researchers at UNC School of Medicine and Duke University demonstrate why coughing often cannot clear mucus from airways in diseases like CF and COPD. They show how to make mucus thinner and less sticky, allowing coughing to become a therapeutic aid for patients.
A study analyzes airway mucus properties contributing to ineffective coughing in respiratory disease. Strategies to reduce mucus hyperconcentration and viscosity are proposed as potential solutions.
Researchers have identified a heterogeneous pattern on liver ultrasounds as a predictor of advanced liver disease in cystic fibrosis patients. Children aged 10 years old are at the average age of diagnosis, and 25% develop advanced liver disease within four years.
A new study found that hypertonic saline improved lung function and weight gain in infants with cystic fibrosis. The treatment, which was safe from diagnosis onwards, also showed promise for preventing the progression of lung disease.
Most cystic fibrosis patients do not achieve high enough antibiotic concentrations in their blood to effectively fight bacteria responsible for pulmonary exacerbations. Real-time monitoring of serum antibiotic concentrations could help improve clinical outcomes and reduce worsening pulmonary function.
Researchers have uncovered the importance of mucus clearance in maintaining healthy airways. In chronic lung diseases, such as cystic fibrosis and COPD, immobile mucus layers can accumulate bacteria, damaging lungs. The studies suggest using inhalation sprays to keep mucus bundles moving may impede formation of these harmful layers.
Researchers discovered that tweaking pH balance and oxygen in a CF lung model can help eradicate pathogenic bacteria while minimizing antibiotic resistance. The study found two distinct communities of microbes in CF lungs: anaerobes thriving in low-oxygen and low-pH regions.
Research shows that babies with cystic fibrosis are born weighing less than those without the condition, even accounting for premature births. The study found a link between CF mutation and intrauterine growth, highlighting the need for further research into improving health outcomes before birth.
Research shows that cystic fibrosis affects the development of babies in the womb, leading to lower average birth weights. The study found that CF can impact birth weight even after accounting for premature birth, suggesting a potential link between CF and intrauterine growth.
A new type of airway cell, pulmonary ionocytes, has been found to express high levels of CFTR, a protein mutated in cystic fibrosis. This discovery provides promising targets for future therapeutic strategies against the disease.
Researchers at the University of Adelaide successfully applied cell transplantation therapy to replace damaged cells in CF patients, providing a potential cure. The innovative method involves harvesting adult stem cells from the lungs, correcting them with gene therapy, and reintroducing them back into the patient.
Researchers identified a rare pulmonary ionocyte cell type in airway tissue, playing a key role in cystic fibrosis. The new cell subtype expresses CFTR at high levels, challenging previous assumptions about airway function.
A team of researchers has identified a new type of pulmonary ionocyte, which is the site of cystic fibrosis-causing CFTR gene activity. This discovery could lead to new therapeutic approaches by increasing CFTR activity in affected cells.
A randomized, controlled trial found that supplemental antioxidants decreased the risk of time to first exacerbation requiring antibiotics in patients with CF. The study also showed increased circulating antioxidant concentrations and transiently decreased inflammation in the antioxidant-treated group.